The TOURMALINE Clinical Development Program - Business Wire

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The TOURMALINE Clinical Development Program: An in-depth investigation of ixazomib in multiple myeloma and AL amyloidosis Multiple myeloma is a rare form of cancer that starts in plasma cells, which are found in the bone marrow.1 It is the second most common blood cancer and represents approximately 1 percent of all diagnosed cancers worldwide.2 Despite treatment advances in the past decade, multiple myeloma remains a devastating, relapsing and incurable cancer. Long-term treatment is emerging as a standard of care in multiple myeloma because it has demonstrated the potential to improve clinical outcomes.3 In addition, proteasome inhibition is an established standard of care in the treatment of multiple myeloma. Ixazomib’s TOURMALINE program is designed to evaluate whether sustained therapy with an oral proteasome inhibitor improves the outcomes for patients living with multiple myeloma and AL amyloidosis. TOURMALINE investigates ixazomib in treatment settings and in combinations that have not previously been studied in Phase 3 clinical trials of proteasome inhibitors. The TOURMALINE program is the result of Takeda’s long standing history of patient-centric innovation in multiple myeloma and the company’s continuing commitment to individual patient needs in populations across the continuum of multiple myeloma care. Takeda would like to express its appreciation for the patients, their families and trial investigators participating in the TOURMALINE program, without whom this would not be possible. TRIAL

DESCRIPTION A Phase 3 Study of Oral Ixazomib Plus Lenalidomide and Dexamethasome in Adult Patients With Relapsed and/or Refractory Multiple Myeloma 4

(NCT01564537)

»» Initiated: August 2012 »» Design: Global, randomized, double-blind, placebo-controlled, multi-center »» Eligibility: Patients with relapsed/refractory multiple myeloma who have received 1 to 3 prior therapies »» Primary Endpoints: Progression-free survival (PFS) »» Enrollment: 722 patients worldwide; Completed »» Status: This study met its endpoint at the first pre-specified interim analysis. Data are expected to be presented by the end of 2015. https://clinicaltrials.gov/ct2/show/NCT01564537

TRIAL

DESCRIPTION A Phase 3 Study of Oral Ixazomib Plus Lenalidomide and Dexamethasone in Adult Patients With Newly Diagnosed Multiple Myeloma 5

(NCT01850524)

»» Initiated: May 2013 »» Design: Global, randomized, controlled, open-label, multi-center »» Eligibility: Patients with newly diagnosed multiple myeloma ineligible for stem cell transplant »» Primary Endpoints: PFS »» Enrollment: Approximately 700 patients worldwide; Open for enrollment https://clinicaltrials.gov/ct2/show/NCT01850524

USO-IXA-15-0018

TRIAL

DESCRIPTION A Phase 3 Study of Oral Ixazomib Maintenance Therapy in Patients With Multiple Myeloma Following Autologous Stem Cell Transplant 6

(NCT02181413)

»» Initiated: July 2014 »» Design: Global, randomized, placebo-controlled, double-blind, multi-center »» Eligibility: Patients with symptomatic multiple myeloma with prior induction therapy and ASCT and no post-ASCT consolidation therapy »» Primary Endpoints: PFS »» Enrollment: Approximately 650 patients worldwide; Open for enrollment https://clinicaltrials.gov/ct2/show/NCT02181413

TRIAL

DESCRIPTION A Phase 3 Study of Oral Ixazomib Maintenance Therapy in Patients With Newly Diagnosed Multiple Myeloma Not Treated With Stem Cell Transplantation 7

(NCT02312258)

»» Initiated: April 2015 »» Design: Global, randomized, placebo-controlled, double-blind, multi-center »» Eligibility: Patients with newly diagnosed multiple myeloma with response to prior induction therapy and not receiving stem-cell transplantation »» Primary Endpoints: PFS »» Enrollment: Approximately 700 patients worldwide; Open for enrollment https://clinicaltrials.gov/ct2/show/NCT02312258

TRIAL

DESCRIPTION A Phase 3 Study of Oral Ixazomib Plus Dexamethasone in Relapsed or Refractory Systemic Light Chain (AL) Amyloidosis 8

(NCT01659658)

»» Initiated: December 2012 »» Design: Global, randomized, controlled, open-label, multi-center »» Eligibility: Biopsy-proven diagnosis of AL amyloidosis that is relapsed or refractory after 1 or 2 prior therapies »» Primary Endpoints: Number of patients with overall hematologic response »» Enrollment: Approximately 250 patients worldwide; Open for enrollment https://clinicaltrials.gov/ct2/show/NCT01659658

For more information, please visit https://www.clinicaltrials.gov/.

1 Palumbo A, Anderson K. Multiple Myeloma. New England Journal of Medicine. 2011;364:1046-60. 2 Mateos MV, San Miguel JF. How should we treat newly diagnosed multiple myeloma patients? Hematology Am Soc Hematol Educ Program. 2013;2013:488-495. 3 Palumbo A, Gay F, Cavallo F et al. Continuous Therapy Versus Fixed Duration of Therapy in Patients With Newly Diagnosed Multiple Myeloma. Journal of Clinical Oncology. 2015;60:2466 4 US National Institutes of Health Clinical Trials Registry. A phase 3 study comparing oral ixazomib plus lenalidomide and dexamethasone versus placebo plus lenalidomide and dexamethasone in adult patients with relapsed and/or refractory multiple myeloma. https://clinicaltrials.gov/ct2/show/NCT01564537. Updated August 4, 2015. Accessed September 1, 2015. 5 US National Institutes of Health Clinical Trials Registry. IXAZOMIB plus lenalidomide and dexamethasone versus placebo plus lenalidomide and dexamethasone in adult patients with newly diagnosed multiple myeloma. https://clinicaltrials.gov/ct2/show/NCT01850524. Updated February 23, 2015. Accessed September 1, 2015. 6 US National Institutes of Health Clinical Trials Registry. A study of oral ixazomib citrate (MLN9708) maintenance therapy in patients with multiple myeloma following autologous stem cell transplant. https://clinicaltrials.gov/ct2/show/NCT02181413. Updated March 31, 2015. Accessed September 1, 2015. 7 US National Institutes of Health Clinical Trials Registry. A study of oral ixazomib maintenance therapy in patients with newly diagnosed multiple myeloma not treated with stem cell transplantation. https://clinicaltrials.gov/ct2/show/NCT02312258. Updated July 26, 2015. Accessed September 1, 2015. 8 US National Institutes of Health Clinical Trials Registry. Study of dexamethasone plus ixazomib or physicians choice of treatment in relapsed or refractory systemic light chain (AL) amyloidosis. https://clinicaltrials.gov/ct2/show/NCT01659658. Updated February 24, 2015. Accessed September 1, 2015.

USO-IXA-15-0018